Glycogen or polysaccharide storage disease treatment method

Patent metadata

Field	Value
Publication number	US-2025082599-A1
Application number	US-202418771018-A
Country	US
Kind code	A1
Filing date	Jul 12, 2024
Priority date	Jul 2, 2004
Publication date	Mar 13, 2025
Grant date	—

Abstract

A method for treating glycogen storage disease by administering an effective amount of a composition that includes ketogenic odd carbon fatty acids that ameliorate the symptoms of these diseases.

First claim

What is claimed is:

1 . A method of treating a fatty acid oxidation disorder (FOD) in a subject in need thereof, wherein the method comprises administering to the subject a composition comprising an active agent, wherein the active agent consists essentially of a triheptanoin, and wherein the composition has an acid value of 0.1 or less mg KOH per gram and a hydroxyl value of 2.8 or less mg KOH per gram.

2 . The method of claim 1 , wherein the composition is administered in a range of 1 to 4 grams of triheptanoin per kilogram body weight per day.

3 . The method of claim 1 , wherein the composition is administered in a range of 0.1 to 2 grams of triheptanoin per kilogram body weight per day.)

4 . The method of claim 1 , wherein the composition is in a dosage unit, wherein the dosage unit comprises at least 15 grams of the triheptanoin.

5 . The method of claim 4 , wherein the dosage unit comprises 15 to 75 grams of the triheptanoin.

6 . The method of claim 1 , wherein the composition is provided in an amount that provides 15% to 40% of the daily dietary calories of the subject.

7 . The method of claim 1 , wherein the composition is provided in an amount that provides 20% to 35 % of the daily dietary calories of the subject.

8 . The method of claim 1 , wherein the composition is provided in an amount that provides 25% to 35% of the daily dietary calories of the subject.

9 . The method of claim 1 , wherein the composition is provided as a nutritional supplement.

10 . The method of claim 1 , wherein the composition is provided as a dietary formulation.

11 . The method of claim 1 , wherein the composition is a pharmaceutical composition.

12 . The method of claim 1 , wherein the composition is administered parenterally.

13 . The method of claim 1 , wherein the composition is administered enterally.

14 . The method of claim 1 , wherein the composition is administered orally.

15 . The method of claim 1 , wherein the FOD is selected from carnitine palmitoyl transferase I (CPT I) deficiency, carnitine-acylcarnitine translocase (CACT) deficiency, carnitine palmitoyl transferase I (CPT II) deficiency, very long chain acyl-CoA dehydrogenase (VLCAD) deficiency, trifunctional protein (TFP) deficiency, long chain acyl 3-hydroxy CoA dehydrogenase (LCHAD) deficiency and short chain acyl CoA dehydrogenase (SCAD) deficiency.

16 . The method of claim 14 , wherein the FOD is CPT II deficiency.

17 . The method of claim 11 , wherein the FOD is VLCAD deficiency.

18 . The method of claim 14 , wherein the FOD is LCHAD deficiency.

19 . The method of claim 14 , wherein the FOD is TFP deficiency.

20 . The method of claim 14 , wherein the FOD is CACT deficiency.

Assignees

• Baylor Res Institute

Inventors

• Roe Charles R

Classifications

• A61K9/0053

  Mouth and digestive tract, i.e. intraoral and peroral administration · CPC title

• A23V2002/00

  Food compositions, function of food ingredients or processes for food or foodstuffs · CPC title

• A23L33/16

  Inorganic salts, minerals or trace elements · CPC title

• A23L33/115

  Fatty acids or derivatives thereof; Fats or oils · CPC title

• C12Y302/0102

  Alpha-glucosidase (3.2.1.20) · CPC title